Extensive rod and cone photoreceptor-cell degeneration in rat models of giant axonal neuropathy: implications for gene therapy of human disease-Reference-Cited by-同舟云学术

Extensive rod and cone photoreceptor-cell degeneration in rat models of giant axonal neuropathy: implications for gene therapy of human disease

Published:2021-05-06 Issue:5 Volume:42 Page:600-603
ISSN:1381-6810
Container-title:Ophthalmic Genetics
language:en
Short-container-title:Ophthalmic Genetics

Author:

Armao Diane¹²^ORCID,Bouldin Thomas W.¹,Bailey Rachel M.³,Gray Steven J.³⁴⁵

Affiliation:

1. Department of Pathology and Laboratory Medicine, University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA

2. Department of Radiology, University of North Carolina School of Medicine, Chapel Hill, North Carolina, USA

3. Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas; Viral Vector Facility Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, Texas, USA

4. Department of Molecular Biology, University of Texas Southwestern Medical Center, Dallas, Texas, USA

5. Department of Neurology and Neurotherapeutics, University of Texas Southwestern Medical Center, Dallas, Texas, USA

Funder

Hannah’s Hope Fund

NIH National Institute of Neurological Disorders and Stroke

NIH National Institute of Child Health and Human Development

Publisher

Informa UK Limited

Subject

Genetics(clinical),Ophthalmology,Pediatrics, Perinatology, and Child Health

Link

https://www.tandfonline.com/doi/pdf/10.1080/13816810.2021.1923036

Reference16 articles.

1. Giant axonal neuropathy: An updated perspective on its pathology and pathogenesis

2. Measuring Disease Progression in Giant Axonal Neuropathy

3. Gene targeting of GAN in mouse causes a toxic accumulation of microtubule-associated protein 8 and impaired retrograde axonal transport

4. Sensory-motor deficits and neurofilament disorganization in gigaxonin-null mice

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1. Intermediate filament dysregulation in astrocytes in the human disease model of KLHL16 mutation in giant axonal neuropathy (GAN);Molecular Biology of the Cell;2023-11-01

2. Intermediate filament dysregulation and astrocytopathy in the human disease model ofKLHL16mutation in giant axonal neuropathy (GAN);2023-03-14

3. Genetic Approaches for the Treatment of Giant Axonal Neuropathy;Journal of Personalized Medicine;2022-12-30